Advocacy

Finding a Drug that's in Development and not Currently Approved

By definition, drugs available for compassionate use are not yet approved by the FDA. The easiest way to get access to an unapproved drug is through a clinical trial being conducted on that drug. When you hear about a new drug, the first step is to see if the patient is eligible for an ongoing clinical trial. If the patient is not eligible for the clinical trial, then it's appropriate to pursue the possibility of compassionate use.

A single listing of unapproved drugs in clinical trials or compassionate use does not exist. Patients and family members most often learn about an unapproved drug being tested for their disease in a number of ways:

Web sites and telephone services

All trials testing effectiveness for cancer are required by law to be listed in www.clinicaltrials.gov. Generally, Phase I and many Phase II trials do not examine effectiveness and may not be listed. NCI's clinical trial listing can be found here.

The most comprehensive telephone service for cancer patients to learn about clinical trials as well as cancer treatment and prevention information is 1-800-4-CANCER (this is the National Cancer Institute's Cancer Information Service).

The National Cancer Institute (NCI) has a real-time online question website. The NCI's general web address is www.cancer.gov. Click on the NCI Live Help button, found on the NCI website search pages. Once you're connected to them, you can type in any question you have — about compassionate use, expanded access or a specific drug — and a trained researcher will help you find answers.

The FDA has established the Office of Special Health Issues, Cancer Liaison Program (301-827-4460), to help cancer patients. You can talk to someone in this office and they will fax, email, or mail you information on how to apply for Single Patient Use and discuss the Expanded Access Programs that are sponsored by drug companies.

You also might find web sites and listservs on the internet where people share information about potential treatments for particular types of cancer. www.ACOR.org is a well-known listserv host site with resources for many different cancers; in addition, many patient advocacy groups offer their own listservs.

News Reports

Because of the many, many outlets for news - both print and electronic - there is much more reporting on health news than there was even five years ago. The quality and depth of these reports ranges from poor to excellent. The inconsistent quality of news reports on a new, unapproved drug can cause confusion for the reader or listener. And if that reader or listener is a cancer patient, the confusion can lead to turmoil and frustration.

For example, a reporter might talk at length about a promising new drug for lung cancer and even interview the patient taking the drug without ever naming the drug or explaining how the listener might find out about where they can get more information about the drug.

When this happens you can call the news outlet (e.g. the network or newspaper reporting the story) and ask to speak to the reporter who wrote the story. The reporter should be able to tell you details about the drug that were not included in his news report.

You can then call the drug company and ask where clinical trials are being conducted or if the company is providing the drug in a compassionate use program.

TIP: Financial publications are often ahead of the curve in pharmaceutical research.

REMEMBER: Always proceed with caution and a healthy dose of skepticism for any touted miracle cure. If it sounds too good to be true, it almost surely is.

Patient's Physician

The patient's physician will often be aware of new treatments and drugs that are being developed for your disease. Ask him/her about the clinical trials that are underway for your disease.

REMEMBER: Physicians rarely have the time to follow-up or do searches for clinical trials for the patient. The patient or family member will need to follow-up on suggestions the physician may have. Keep in mind, that physicians have varying views on clinical trials and compassionate use. Their opinion is important but the patient makes the ultimate decision about treatments.

Patient Advocacy Organizations

Our Cancer Resources section contains a list of cancer advocacy organizations. Some advocacy organizations maintain a list of drugs in development on their websites, along with information about current research.

Medical Journal Articles

If you're good at deciphering medical lingo, you can search for medical journal articles about treatments for a particular type of cancer, or for those about a specific medication. The National Library of Medicine's (NLM) database, which includes millions of articles, is at www.nlm.nih.gov. The specific address for searching the database is www.ncbi.nlm.nih.gov/pubmed.

Many times the NLM's database will provide the full text of the article you are interested in but often it will only be an abstract or summary of the article. Obtaining the full article may require a visit to a medical library or payment of a fee.

Your local public library's system might also allow you to search on various medical websites not ordinarily available to the public without a charge.

Remember — most pharmaceutical companies DO NOT provide their unapproved drug outside of the clinical trials that they are conducting on their drug.